For years, the Food and Drug Administration has struggled with medications that are approved for market but later turn out to have serious side effects its reporting system failed to spot.
Now, a proposal that could be a big step toward solving the problem has been offered by no less an authority than the head of the federal Medicare system. But the response of FDA officials seems lukewarm at best.
Whether it’s a suspected link between Viagra and rare cases of vision loss or heart attacks among arthritis patients using Vioxx, the FDA’s current system of voluntary reporting picks up no more than 10% of serious drug reactions.
To help shrink the knowledge gap, Medicare head Mark McClellan, a doctor and an economist who once ran the FDA, has proposed a Medicare-based system to track the safety and effectiveness of medications.
McClellan proposes taking billing data from the prescription program, which will begin in January, and combining it with healthcare information already collected when Medicare users submit claims for hospital and doctors’ care. By cross-referencing the information, a computer system could spot signs of trouble.
Patients’ personal information would be protected, but the data pool would be so large that problems could be much easier to detect than they are now, he said.
The problem now is that the FDA approves drugs for market on the basis of trials involving a few thousand patients -- and sometimes dangerous side effects don’t show up until a prescription is widely used on the open market.
The Medicare data would cover millions of patients and thus create an important warning network.
“One of the main frustrations with Vioxx was that it was on the market for a number of years and used by millions of people, and only later was there a possible association with problems,” McClellan said in an interview.
“There is a legitimate reason for the public to be concerned. In an era when we should be depending on electronic systems, it should not take five years and millions of patients before we get to a potential problem.”
The FDA, however, has been noncommittal. “It really is too early,” a spokeswoman said. “The agencies are exploring this possibility.” The FDA declined to make senior medical officers who oversee drug safety available for interviews.
Some outside advisors to the FDA are baffled by what they see as a lack of resolve. The Medicare-based system was one of several ideas discussed at a recent FDA drug safety advisory committee meeting on ways to improve monitoring and reporting.
But after two days of deliberations, “no one at the agency was willing to say, ‘OK, this is what we would like to do,’ ” said Dr. Curt Furberg of Wake Forest University, a member of the advisory panel.
“We thought we had some good ideas, and [using Medicare data] was one of them. It’s a little bit frustrating. I really don’t understand what their problem is.”
The panel’s chairman, Dr. Peter Gross of Hackensack University Medical Center, said he thought the Medicare proposal was a good idea, but he “didn’t get the sense that [FDA officials] were latching on to it.”
“It takes them a long time to come up with decisions,” he added.
A large private insurer, UnitedHealth Group Inc., is already creating a monitoring system similar to the one McClellan has proposed. The UnitedHealth system could cover as many as 20 million of its patients; the Medicare plan could cover as many as 43 million people, including some of the sickest and most vulnerable, whom drug companies don’t usually enroll in clinical trials.
The prospect of using Medicare to monitor drug safety has generated a buzz of interest among medical and pharmaceutical experts.
“I think it’s a huge opportunity,” said Dr. Raymond Woosley, vice president of the University of Arizona Health Sciences Center. “We’re going to be spending billions of dollars on medications for the elderly, and we’re calling it a ‘benefit.’ But if we don’t have a way to monitor outcomes from the use of these medicines, it may not be a benefit.”
Woosley, who has testified before Congress on ways to improve drug safety, said he believed the FDA would ultimately endorse the idea.
The centerpiece of the agency’s current safety surveillance network is a database called the Adverse Event Reporting System. Doctors and hospitals voluntarily submit reports, and drug companies are required to pass on the ones they receive. Although the system collects about 400,000 reports a year, most experts believe it only scratches the surface.
“We don’t know the true extent of underreporting, but we know it’s vast,” Joyce Weaver, a pharmacologist with the FDA’s office of drug safety, told the advisory committee.
The reporting system is considered effective for spotting rare side effects, such as sudden liver failure. But it is much less reliable for distinguishing whether a drug caused a relatively common medical problem such as a heart attack. It also tends to miss risks that emerge after the first few years of a drug’s use, when a medication’s novelty has worn off.
Moreover, because it is a collection of individual reports, the FDA’s system doesn’t give scientists any idea of the total number of patients taking a drug -- a key piece of information for calculating the prevalence of any problem.
A Medicare-based surveillance system would address each of those shortcomings. Reporting would be automatic. It would continue year after year. And it would provide a better sense of the total number of patients taking a medication.
The UnitedHealth system, set to launch in August, will track the four new drugs most frequently prescribed for UnitedHealth patients: the erectile dysfunction drug Cialis, the pulmonary drug Spiriva, the antibiotic Ketek and the antidepressant Cymbalta.
The health outcomes of patients taking each of the new drugs will be compared with results for patients using the current leading drug prescribed for the same medical problem.
Dr. Alec Walker, senior vice president for epidemiology at Ingenix Inc., the UnitedHealth division that devised the system, said the goal is to track all new drugs that have at least 1,000 users.
“What we are doing is the obvious thing someone should be doing, and the same applies for Medicare data, which will be as rich as ours,” Walker said. “It’s self-evident to anyone who really thinks about the data resources and is looking for a systematic way to do things.”
Because taxpayers will already be paying for the Medicare information and the computers that would crunch the data, there would be very little cost associated with setting up and operating the system.
“The electronic systems we would need are already going to be in place,” said McClellan, the Medicare administrator. “We need them for Medicare to determine what to pay. The incremental cost of doing this is very low ... well under $10 million a year.”
In Congress, Sens. Charles E. Grassley (R-Iowa) and Max Baucus (D-Mont.) are considering legislation to authorize Medicare to set up the monitoring system. McClellan says his interest is broader than safety: He wants to compile hard data on what kinds of drug treatments are most cost-effective.
Efforts to bring such transparency to the pharmaceutical marketplace are in their infancy.
“Some people may be afraid of this data,” said Henri R. Manasse Jr. of the American Society of Health-System Pharmacists. “If this system were to detect a lot of bad prescribing related to the marketing practices of the pharmaceutical industry, the industry would not be very happy. So there could be some political downside, but medically it could help deliver the best care for the available money.”
Dr. John Santa, assistant director for health projects at the Center for Evidence-Based Policy at Oregon Health & Science University, said the Medicare data could prove very valuable to doctors and patients trying to sort through the claims of drug makers.
“It would be incredible if this resulted in a cadre of researchers free from the influence of the industry,” said Santa. “The country would be much better served.”
But he said the government shouldn’t limit its interest to Medicare patients only.
“Why aren’t they doing the same thing for millions of children covered under Medicaid?” Santa asked. “Such a registry could be extraordinarily helpful in identifying problems.”